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IAMRARE™ Registry Program

Rare diseases present unique challenges for researchers and companies working towards treatments and cures:

Small patient populations; Hard to diagnose or delayed diagnosis due to lack of education and understanding among medical professionals; Poor understanding of the natural history of the disease and its progression without intervention; No prior pathways to follow; Clinical endpoints that are often unclear; and enrollment and retention challenges.

To address the special needs of those developing treatments for rare diseases, NORD has created a Natural Histories Patient Registry Platform, with extensive input from FDA, NIH, patients, organizations and experts in the field.

NORD’s platform is an easy to use system that allows patients and organizations to inform and shape medical research and translational science for rare diseases by launching high-quality, customized registries to collect the data needed to define the natural progression of their disease – ultimately advancing product development.

 

30aTo learn more about this program, please contact us using the form below.

Recent Updates

Press Release: NORD Announces 20 Rare Disease Groups Selected to Develop Natural History Studies as Part of FDA Cooperative Agreement. Read the full release here >

FDA Senior Official says NORD’s platform is “an exciting new tool researchers are using to study rare diseases.” Read the blog here >

BioCentury writes article Charting Rare Stars: How NORD is putting patients in control of rare disease natural history studies. Read the article >

The Power of Patients

In 2019, NORD collaborated with Trio Health on a book based on natural history data. The Power of Patients: Informing Our Understanding of Rare Diseases is based on natural history data collected by NORD’s registry community and features a foreword by Janet Woodcock, MD, Director of FDA’s Center for Drug Evaluation and Research.

The book amplifies the experiences of individuals and families living with rare diseases and demonstrates the importance of registries and natural history data in helping to shape the rare disease landscape. By sharing stories, celebrating community-driven teamwork, and exhibiting data from NORD’s registries, The Power of Patients aims to illustrate the promise and potential of longitudinal natural history studies.

To request a full digital copy or printed version, please email research@rarediseases.org. Otherwise, you may download digital chapters below.

Introduction 

Chapter 1 – OMS 

Chapter 2 – PKU

Chapter 3 – Pemphigus and pemphigoid

Chapter 4 – SYNGAP1

Chapter 5 – Desmoid Tumor

Chapter 6 – ITP 

Posters and acknowledgments 

NORD Chairman, Marshall L. Summar, MD, on the need for registries in rare diseases

Originally published on Medscape Rare Diseases as part of the
NORD and Medscape Editorial Collaboration.

Features

• NORD’s Registry platform utilizes a cloud-based, modern design that is mobile-friendly, safe and easy to use

• A dedicated IAMRARE team offers ongoing support and guidance – from planning, to production, and beyond

• Smart surveys target question participation and enable natural history data tracking

• Study resources include standardized data dictionaries and access to rare disease IRB expertise

• Automated survey reminders encourage long-term user engagement

• Role-based permissions allow for flexible study design

• Analysis tools provide users with real-time data for comparison to other patients

FDA Cooperative Agreement

i1In 2015, the FDA awarded NORD with a $250k grant to build 20 new registries.

                        Read More >

Additional Resources

NORD has assembled the following resources and links to help patients, organizations, researchers and  companies interested in rare disease registries and natural history studies.

In 2019, NORD collaborated with Trio Health on a book based on natural history data. The Power of Patients: Informing Our Understanding of Rare Diseases is based on natural history data collected by NORD’s registry community and features a foreword by Janet Woodcock, MD, Director of FDA’s Center for Drug Evaluation and Research.

The book amplifies the experiences of individuals and families living with rare diseases and demonstrates the importance of registries and natural history data in helping to shape the rare disease landscape. By sharing stories, celebrating community-driven teamwork, and exhibiting data from NORD’s registries, The Power of Patients aims to illustrate the promise and potential of longitudinal natural history studies.

To request a full digital copy or printed version, please email research@rarediseases.org. Otherwise, you may download digital chapters below.

Introduction 

Chapter 1 – OMS 

Chapter 2 – PKU

Chapter 3 – Pemphigus and pemphigoid

Chapter 4 – SYNGAP1

Chapter 5 – Desmoid Tumor

Chapter 6 – ITP 

Posters and acknowledgments 

http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm458485.pdf

http://www.effectivehealthcare.ahrq.gov/ehc/assets/File/Patient-Powered-Registries-white-paper-130911.pdf

http://www.tandfonline.com/doi/pdf/10.1517/21678707.2015.1063415

https://events-support.com/Documents/Pariser.pdf

http://effectivehealthcare.ahrq.gov/ehc/products/420/1897/registries-guide-3rd-edition-vol-2-140430.pdf

https://rarediseases.org/wp-content/uploads/2015/12/EUCERD_Recommendations_RDRegistryDataCollection_adopted.pdf

Interested in a registry? Contact us.